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New Bio Fund Partner Describes A16Z Investment Strategy

Wed, 11/25/2015 - 12:08

When Marc Andreessen and Ben Horowitz founded their namesake venture firm in 2009, they laid out a clear-but-narrow vision for investing in a new wave of Web-based innovation.

Andreessen, in particular, espoused a net-centric view that was absolute. “No clean tech, no rocket ships, no electric cars. No China or India,” he told Fortune magazine at the time. Biotech likewise was out of the question. In the six years since then, Andreessen Horowitz has grown into a $4 billion VC, and established itself as a leading tech investor. Andreessen’s observation that “software is eating the world” has become an industry axiom, as Web-based services have invaded and taken over financial services, education, and a host of other sectors.

Now the venture firm also known as A16Z has formed a new $200 million fund to make software investments at the intersection of tech and life sciences, where software companies are developing new ways analyze and process data for biotech and healthcare companies. Money for the firm’s new bio-fund was all new, raised from existing limited partners and not by carving out funding from A16Z’s flagship venture or opportunities funds.

One of the firm’s first investments is TwoXar, a Palo Alto, CA-based tech startup using proprietary algorithms to analyze biological data in both public and proprietary databases with the goal of identifying previously unknown effects of drug candidates in early stage discovery. The idea is to point the way to compounds that could be developed as new drugs for metabolic and neurological disorders.

TwoXar “fits our investment thesis in that they use machine learning and cloud biology,” said Vijay Pande, who joined Andreessen Horowitz as a general partner dedicated to the bio fund.

Vijay Pande

Pande has defined “cloud biology” as much like cloud computing. In addition to providing computational services, though, Pande has said that Web-based programs also can be used to carry out automated experiments more precisely and consistently than lab technicians (enabling biotech companies to optimize the reproducibility of their scientific results).  At Stanford, where Pande was previously a scientist, his lab specialized in … Next Page »

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At Duchenne Panel, Parents Plead With Experts, FDA Decision Looms

Wed, 11/25/2015 - 00:20

In one of the more dramatic biotech events in recent memory, the FDA convened an advisory panel on Tuesday to weigh the risks and benefits of a drug called drisapersen, which has the chance to be the first FDA-approved treatment for Duchenne muscular dystrophy—a progressive, fatal genetic disease.

The panel’s task was unusual, as far as these meetings go. Typically, panel members are asked whether they’d recommend approval of a drug. The agency then takes the panel results under consideration when it issues its final decision later on.

In this case, however, the agency did not ask the panel members for a yes or no recommendation. Instead, panelists were asked to assess the strength of the evidence accrued in the clinical trials of drisapersen, a Duchenne drug from San Rafael, CA-based BioMarin Pharmaceutical (NASDAQ: BMRN).

The panelists did so, and their commentary did not seem favorable, which stood out all the more because of the emotional pleas for the drug’s approval that came from many of the parents of Duchenne patients—and the patients themselves—who showed up at the meeting in Silver Spring, MD.

“Our children are fighting for their lives and they deserve therapies that can change their quality of life, and the outcome of their disease,” said one parent. “This should be the day that the ending of this tragic story is rewritten…please approve this drug.”

The panelists voted on whether the data from each of three different placebo-controlled clinical trials strengthened, weakened, or had no effect on BioMarin’s case for approval. They then wrapped up the panel by discussing—but not voting on—the drug’s benefits versus its risks.

By most accounts, it’s very unusual for the agency not to ask advisory panels for a yes-or-no vote about a drug. RBC Capital Markets analyst Michael Yee called this omission “unlike most panels” in a research note, and Leerink Partners’ Joseph Schwartz deemed it “unique and leaves the FDA open to do its own thing.” The FDA will make a decision on BioMarin’s drug by Dec. 27.

When asked why FDA did not want such a vote on drisapersen, FDA spokeswoman Sandy Walsh wrote in an email, “We want to gain input from the panel and will engage in substantive discussions today and discuss strength of evidence.”

When asked how often FDA tells an advisory panel not to weigh in with a yes-or-no vote on a drug, Walsh said FDA doesn’t keep track. Setting the questions up this way, Leerink’s Schwartz wrote, “should alleviate some of the intense pressure on [the] panel to reach overall conclusions.”

The FDA usually follows the guidance of its advisory panels, but not always. Since there wasn’t any voting directly for or against drisapersen—just on the relative strength and persuasiveness of the data—that guidance will be harder to read.

But the majority of panelists did vote that two of BioMarin’s studies “weakened” its case for approval. What’s more, some of them directly said they didn’t think drisapersen should be approved.

Justin Zivin, a professor from UC San Diego, noted that after reading the FDA’s briefing materials on drisapersen, “it was clear that this wasn’t going to get approved at this point. This just needs more work.”

Added Chiadi Onyike, a professor at Johns Hopkins: “What we have in front of us…is not yet ready.”

Still, this is a very complex issue. Duchenne patients, and parents of patients, are desperate for a drug for a deadly disease with no approved treatment. The drisapersen panel is as close as they’ve been to an FDA approved therapy, and many parents—and some young patients themselves—came to the hearing to make an impassioned plea for the drug before the panelists. One 16-year-old boy, for instance, claimed drisapersen had helped him walk longer without getting tired, and gave him the strength to swim and ride a bike. “Please don’t take this drug away from us,” he said. A round of applause followed.

But drugs are approved on empirical data, not powerful anecdotal evidence, and there are significant questions regarding BioMarin’s data. The key study of drisapersen, a Phase 3, 186-patient, placebo-controlled study, failed. It’s unclear whether the drug—as it’s designed to do—helps Duchenne patients produce enough dystrophin, the protein they lack, which normally helps keep muscles intact.

BioMarin is making the case that the study was flawed, that patients were enrolled too far along in their disease, and that a post-hoc analysis pooling data from subgroups of patients shows drisapersen does in fact help Duchenne patients. The totality of the data, BioMarin says, shows that there is a signal.

Today’s meeting on drisapersen will also likely affect the fortune of a rival drug from Sarepta Therapeutics (NASDAQ: SRPT), eteplirsen, that will be the subject of an FDA advisory panel in January. As with BioMarin, Sarepta’s case will also be flawed. It doesn’t have nearly the amount of data that BioMarin has on drisapersen. Even though the drug has so far looked safer than drisapersen, side effects could emerge as more patients are tested. When the FDA releases briefing documents for eteplirsen in the days leading up to the panel in February, new questions could face Sarepta as well.

Duchenne is a disease with no cure and no effective treatment. It sentences the roughly 300,000 boys who have it—the disease mostly affects boys—to a slow, progressive decline in muscle function. Many lose the ability to walk by their teens and die at a young age from one of a number of complications, such as respiratory failure.

Ilan Ganot is the father of a son with Duchenne and founded a company, Solid Biosciences, to fight the disease. Ganot told Xconomy recently, “This is a battle against time.” And that’s what made Tuesday’s panel so critical a point in the Duchenne battle. Every single FDA move either brings patients closer, or further away, from a potential treatment. Should the agency ultimately reject drisapersen, patients and their families will watch more time pass before a drug is available.

The severity of Duchenne and the significant need for treatments by nature lowers the bar for a drug’s safety and effectiveness. That’s why BioMarin paid … Next Page »

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Small Molecules: The Silent Majority of Pharmaceutical Pipelines

Mon, 11/23/2015 - 05:00

The pharmaceutical industry is constantly evolving. There always seem to be new, innovative therapeutic approaches emerging that capture our imagination and advance our ability to make a difference in the lives of patients. Take, for example, T-cell therapy for cancer, microbiome therapies, and CRISPR-Cas9 gene editing, each of which could potentially have a significant impact on healthcare someday.

Yet despite these and other advances, I expect one thing won’t change: small, synthetic molecules will remain remarkably prevalent in both development pipelines and on the market. I think of them as a silent majority. They might not be grabbing the headlines, but they consistently deliver results.

This might sound surprising. After all, we’re talking about good old-fashioned organic chemistry with drugs that are made in chemical reactors rather than in cells or living tissues. But consider some of the following points before relegating these small molecules to obscurity.

Small molecule drugs still account for about 90 percent of the therapeutics in today’s pharmaceutical market. According to Medscape, in 2014 five of the top ten selling drugs were small molecule drugs: aripiprazole (Abilify, $7.2 billion), esomeprazole (Nexium, $6.3 billion), rosuvastatin (Crestor, $5.6 billion), fluticasone propionate (Advair Diskus, $5.0 billion), and sofosbuvir (Sovaldi, $4.4 billion). Nine of the top 10 (and 17 of the top 20) most prescribed drugs in the U.S. last year were small molecules. Medicare lists fluticasone and rosuvastatin as two of its top prescription drugs.

How are these numbers possible? How could small molecules still be so dominant and prevalent despite the emergence of ever-newer, ever more complex cutting-edge therapies? For one, we’re talking about an incredibly, almost unimaginably, diverse group of small chemical compounds that often share nothing in common with one another except for their small molecular size and the fact that they’re made from synthetic chemical reactions.

Just how diverse is this super-family? Consider three of them: aspirin, corticosteroids and ivacaftor (Kalydeco).

Aspirin is probably one of the oldest and most widely used therapeutics in the world, first sold under that brand in 1899. Nowadays, the world consumes more than 40,000 tons of aspirin every year. You can buy a bottle of 100 pills for $1 at your local pharmacy. And yet the potential benefits of aspirin are regularly in headlines as more and more uses continue to emerge. Aspirin is now used for a range of indications, from cardiovascular health, to Alzheimer’s disease, cancer treatment, pulmonary diseases, and of course, general everyday aches and pains. Amazingly, Bayer aspirin generated an estimated $998 million in sales worldwide in 2014. This for a drug that was originally patented under the Kaiser!

Then there are corticosteroids (such as cortisone), another example of a decades’ old, ubiquitous anti-inflammatory drugs. These drugs were first synthesized in 1949, and it’s almost impossible to think of any human on the planet who hasn’t used them at least once. These drugs are among the most potent anti-inflammatory drugs around (they mimic a hormone the body makes called cortisol) and can be formulated into almost any imaginable dosage; be it oral, topical, or injectable. Sadly, their side effects are often just as potent as their benefits, making them ill-suited for chronic therapy.

But “small molecule” doesn’t necessarily mean old drug. We see them featured in the newest, most cutting edge drugs; often tailored to fit very specific genetic subsets of patients.

A prime example is Vertex Pharmaceuticals’s ivacaftor. This is the first drug approved (in 2012) to treat a specific malfunctioning protein caused by the genetic defect in cystic fibrosis patients. Ivacaftor is currently approved for six percent of the 70,000 patients with CF worldwide. With its annual price of over $300,000, analysts estimate that ivacaftor will top $1 billion in sales by 2018 and possibly three to five times that when combined with other drugs to boost its reach.

These are just a few examples of the vast number of diverse types of small molecule drugs already on the market (let alone those currently in development). Here are a few reasons why I’m betting that small molecules will continue to dominate pharmaceutical pipelines:

1) It’s almost impossible to imagine just how many small molecules there are out there. Simply using the category “small molecules” is a misnomer. It lumps together molecules whose structure, function, medical usage, potency and safety profiles can be radically, and wonderfully, different.

2) Much of our biology is driven by small molecules, so it makes sense to use another small molecule to address any biological problems. For example, as a rule of thumb, nature likes to use “large molecules” such as proteins and sugars as building blocks and “small molecules” (such as cyclic AMP, which activates enzymes called kinases) for signaling and inhibition.

3) They’re relatively easy to make, scalable, and highly profitable. These are significant advantages. On average, a small molecule drug costs about $1/day to produce, with generics typically costing even less. By comparison, a biologics’ average cost is approximately $22/day or roughly $10,000/year.

Given all this, it shouldn’t come as a surprise that as we look at the therapeutic pipelines of pharma, many companies still continue to develop small molecules for almost every imaginable indication. Pharma giant Roche, after all, recently announced plans to restructure its small molecule manufacturing plant, a definitive nod to the significance of small molecule drugs.

So, with all due respect for the latest advances in our field from antibodies to genetic therapies, I’m going to stick my neck out and posit that this diverse, chaotic, illogical, and huge family we call “small molecules” will continue to play a significant role in the pharmaceutical industry for the foreseeable future.

This silent majority still has a lot to say.

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Paris Rampage Rekindles Conflict Over Government-Proof Encryption

Fri, 11/20/2015 - 20:11

The recent terrorist attacks in Paris were executed with guns and bombs in the physical terrain of city streets and cafe terraces. But the horrific incident is reviving government calls for greater control of the cyber environment.

A host of top U.S. officials including the directors of the CIA and FBI, Senator Dianne Feinstein, New York Mayor Bill de Blasio, and Manhattan District Attorney Cyrus Vance, Jr., have been citing the Paris atrocities to bolster their arguments that tech companies need to give law enforcement ways to break through the impenetrable encryption installed in the latest mobile phones, devices, and apps such as WhatsApp. Their claim is that governments need these powers to prevent terrorist operations, although substantial evidence has yet to emerge that encrypted communication played an important role in mounting that the Paris attack.

Silicon Valley has counterattacked against the government salvos with a war of words this week, through publications including Wired, Re/code, and Gizmodo. Privacy advocates, civil rights leaders, and some tech companies maintain that governments will only do harm if they force device and app makers to create backdoors in their encryption code for crime investigators. They argue that hackers will readily find and exploit those entry points, to more easily steal sensitive personal, corporate—and government—information.

How should businesses react to these counterclaims as they try to protect their data, as well as their physical locations, in an era of increasing cross-border threats?

To get some answers, I touched base this week with a cybersecurity expert who is well versed in the interests of both government agencies and private companies. Mark Weatherford just joined Mountain View, CA-based information security company vArmour as chief cybersecurity strategist. He’s a former cybersecurity deputy with the Department of Homeland Security, and former top security official for two states—California and Colorado.

Weatherford (pictured above) says he understands why law enforcement agencies are frustrated that they can’t decipher the electronic messages of people suspected of criminal and terrorist plots. “That’s a legitimate concern,” he says. But he also sees the downside of creating vulnerable spots in encryption software so investigators can crack the code. “Creating a back door or path for law enforcement is going to open back doors for bad guys, too,” Weatherford says.

In addition to calls for law enforcement access to encrypted messages, Congress is mulling a bill to encourage private companies, including device and app makers, to share cyber threat information with the government. The Cybersecurity Information Sharing Act (CISA), passed by the Senate in late October, would grant companies that cooperate with the government immunity from lawsuits for failing to protect their customers’ privacy. The Electronic Frontier Foundation charges that the law creates “aggressive spying authorities,” includes too few privacy safeguards, and won’t solve the cybersecurity defects that led to data breaches at Target and the U.S. Office of Personnel Management (OPM) this year.

Weatherford says some companies favor the law because of its liability shield, but others don’t want to share anything with the government. “Some companies don’t trust the government at all,” he says.

Although information sharing is critically important, government officials may have a mistaken view that if they get the access they want, “all their problems are going to go away,” Weatherford says. That couldn’t be further from the truth, he says.

The “bigger security pie” must be composed of the right tools, training, and people, Weatherford says.

Another essential element is trust—and the U.S. government has lost some ground on that score.

The American public was stunned when Edward Snowden’s leaks revealed sweeping government data-gathering on ordinary citizens’ communications. Some tech companies have been increasingly resistant to disclosing their customers’ data at government request. Now they maintain that with their current encryption features, even they can’t unlock the meaning of a customer’s messages.

Large American companies will contact the government if they themselves come under a cyberattack, Weatherford says. But there’s still a concern that when a company shares technical information about a malware invasion, for example, the government may also acquire sensitive information about executives, employees, customers, or corporate activities. Suspicion persists that government investigators may share that information more broadly with other U.S. agencies such as the IRS or fraud prosecutors, he says. Companies worry that an appeal for government cybersecurity help might end in an employee’s investigation for tax evasion or drug dealing, he says.

“Unfortunately, the government hasn’t done a good job in proving people wrong when they say it will do what it’s not supposed to do,” Weatherford says. “They don’t have a good track record.”

Even leaked evidence of conduct that’s legal, but distasteful, on the part of top executives could damage a company’s reputation or sink its stock value, Weatherford says. He points to the flood of Sony executives’ e-mails that were spilled online in 2014 after hackers got hold of them.

Another worry for businesses is that government agencies themselves have been repeatedly hacked, and any of their proprietary data stored on government servers could be plundered by … Next Page »

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West Coast Bio Roundup: Shkreli, T4 Diabetes, Cella-Done, A16Z+Bio

Fri, 11/20/2015 - 09:42

So much for lying low. Investors led by Martin Shkreli have bought a majority of the shares of failed South San Francisco, CA-based KaloBios Pharmaceuticals, which has been in dire straits for some time after clinical failures and the abrupt departure of its longtime CEO David Pritchard earlier this year. For those of you just back from a six-month Antarctic expedition, Shkreli became the poster boy for drug industry greed earlier this year when his Turing Pharmaceuticals took over a drug to treat parasitic infections, often used by HIV patients, and raised its price 5,000 percent to $750 a pill.

Our former Xconomy colleague Arlene Weintraub details in Forbes Shkreli’s plans to revive one of KaloBios’s antibody-based drugs to fight the rare cancer CMML, or chronic myelomonocytic leukemia. The firm has tried to direct its antibody-based drugs against asthma, cancer, and infectious disease.

Former CEO Pritchard once told Xconomy that KaloBios had a chance to be a billion-dollar business. If Shkreli can make that come true, he’ll yet again be the industry’s center of attention. Let’s get to the rest of the roundup.

—Researchers at the Salk Institute in San Diego say they’ve gained insight into a type of diabetes that seems to strike elderly people who don’t have the weight gain associated with type 2 diabetes, the most prevalent form of the disease. In mice with a version of this diabetes—the researchers call it “type 4″—they found amongst the fat cells an abundance of immune cells called T regulatory cells, or Tregs. Instead of losing weight, the researchers say, the key to fighting type 4 diabetes could be losing Tregs. They plan to continue the work with fat samples from older, lean people—the kind you can practically scoop up by the dozens as they walk along the beach in Southern California. The paper was published Wednesday in Nature.

—Silicon Valley venture firm Andreessen Horowitz—or a16z in Valleyspeak—has raised a separate $200 million fund to invest in companies at the intersection of computer science and biology. To run the fund, the firm has tapped longtime Stanford University professor and polymath Vijay Pande. One of his projects was Folding@Home, an attempt to use a volunteer network of computers, working in their spare time, to solve exceedingly difficult protein biology problems. As Pande explained in this a16z blog post about the new fund, the declining cost of computational power has made that kind of solution seem quaint in a few short years, and it has also opened up new avenues for solving health problems with software. One area of focus for the new fund will be digital therapeutics, which Xconomy has covered in stories like this one.

—San Diego’s Celladon (NASDAQ: CLDN), which saw a crucial gene therapy trial fail earlier this year, is merging with Palo Alto, CA-based Eiger BioPharmaceuticals. If approved by Celladon’s shareholders, Eiger’s equity investors would become majority owners and the combined entity would take on the Eiger name. An investor syndicate has agreed to invest $39.5 million in the combined company, which would have a pipeline of drugs targeting four rare diseases: Hepatitis D, bariatric surgery-induced hyperinsulinemic hypoglycemia, pulmonary arterial hypertension, and lymphedema. As Ben Fidler reported yesterday, this type of white flag deal happens every so often in biotech.

—In related news, Paul Cleveland, who had been running Celladon up until the Eiger deal, has just been named the new CEO of Avalanche Biotechnologies (NASDAQ: AAVL), of Menlo Park, CA. Like Celladon, Avalanche also had a big miss in a gene therapy trial this year.

—D. Wade Walke, the top communications official at San Diego’s Isis Pharmaceuticals (NASDAQ: ISIS), told CNN that the company was mulling a name change after the Paris attacks perpetrated by the terrorist group that (sometimes) shares the same name. Despite no corporate news, good or bad, and a general Wall Street rally, shares in Isis dropped 4 percent to start the week, prompting the company to wonder if its name is having a negative effect.

—South San Francisco, CA-based diagnostic maker Veracyte (NASDAQ: VCYT) has appointed San Diego biotech veteran Tina Nova to its board of directors.

—San Diego’s Epic Sciences agreed to work with the University of Pennsylvania’s Abramson Cancer Center to identify biomarkers in circulating tumor cells (CTCs) that could predict response to cancer therapies.

—Thermo Fisher Scientific (NYSE: TMO) of Carlsbad, CA, said it is collaborating with Novartis and Pfizer on a companion diagnostic test that the pharma companies plan to use to help develop drugs for non-small cell lung cancer.

—San Diego-based Otonomy (NASDAQ: OTIC) is enrolling the first patients in a Phase 3 clinical trial of OTO-104 for Ménière’s disease, an inner ear disorder characterized by severe dizziness, vertigo, and gradual hearing loss. The company expects to enroll roughly 160 patients in the trial at clinical sites throughout the U.S.

—South San Francisco-based Threshold Pharmaceuticals (NASDAQ: THLD) said it has extended a licensing deal for the cancer drug evofosfamide with Germany’s Merck KGaA to include an option for Threshold to co-promote the drug in the U.S. if it comes to market. Evofosfamide is currently in Phase 3 trials.

—Chris Rivera, who recently ended his stint at the head of Washington state’s biotech trade group, has joined the advisory board of Seattle-based Stratos Group, a life science product development firm.

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Salk Recruits Nobel Laureate Liz Blackburn as First Woman President

Thu, 11/19/2015 - 17:08

The Salk Institute has named Elizabeth Blackburn, a Nobel laureate and UC San Francisco professor of biochemistry and biophysics, as its first woman president.

Blackburn has been a non-resident fellow at Salk since 2001, and will take over as president on Jan. 1. She is succeeding William Brody, a doctor and biomedical engineer who joined the prestigious biomedical research institute in 2009, after serving 12 years as president of Johns Hopkins University in Baltimore, MD. Brody brought financial stability to the Salk, and nearly doubled the institute’s endowment to $356 million.

Blackburn, 66, won the Nobel Prize in Physiology or Medicine in 2009 for elucidating the role of telomeres, the repetitive nucleotide sequences at the tips of chromosomes, and for discovering telomerase, an enzyme that replenishes telomeres. Her work brought new understanding about the function of telomeres in reproduction, and how telomeres play a central role in the effects of chronic stress, aging, and diseases like cancer.

Blackburn joined the faculty at UCSF in 1990 and served as chair of the microbiology and immunology department from 1993-99. In addition to the Nobel Prize, she has received nearly every major award in science, including the Lasker, Gruber, and Gairdner prizes.

Elizabeth Blackburn

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Waving White Flag, Celladon Merges With Rare Disease Co. Eiger Bio

Thu, 11/19/2015 - 10:35

A slew of companies have gone public amidst gene therapy’s recent renaissance, and technological advances have gotten these types of treatments closer than they’ve ever been to impacting healthcare in the U.S.

But today, Celladon offered a reminder of how quickly it can all go wrong in gene therapy. The San Diego biotech and its once promising treatment for a genetic form of heart failure will now effectively disappear, thanks to a merger with a privately held company called Eiger BioPharmaceuticals.

Celladon (NASDAQ: CLDN) has agreed to a deal with Eiger that, if approved by shareholders, would see Eiger’s stockholders become the majority owners of the combined company. The entity would carry forward the Eiger name, and the Palto Alto, CA-based company’s strategic plan to develop treatments for a variety of rare diseases like lymphedema, pulmonary arterial hypertension, and hepatitis D. All of Celladon’s directors and executives will resign from their current positions once the deal is done. The new company will be run by Eiger’s team, which is lead by president and CEO David Cory, and be based in the Bay Area.

This is the type of deal that often happens after a publicly held biotech waves the white flag. In January, for instance, Regado Biosciences of Basking Ridge, NJ, merged with Tobira Therapeutics after safety concerns doomed its potential anticoagulant drug. The combined company is known as Tobira, is one third owned by Regado’s old shareholders, and is now developing drugs for inflammatory and liver diseases. A few years ago, a pain drug developer called Zalicus failed a big trial, and merged with a biosimilars company called Epirus Biopharmaceuticals. Targacept, after a string of clinical failures, merged with Catalyst Biosciences in August. These deals give shareholders of the failed biotech a chance to recoup some value after things go wrong—and a quick way for a private company to tap into the public markets.

Now here’s Celladon, which was a high-flying gene therapy company not too long ago. For those unfamiliar, gene therapy is a way of shuttling genetic instructions into the body via a virus, offering the potential for a long-lasting or even permanent fix for a genetic disease. That promise has tantalized scientists for decades, but as with any new science, the field has gone through a number of ups and downs trying to figure out the best way to safely and effectively deliver these treatments.

The progress of companies like UniQure (NASDAQ: QURE), which has the only approved gene therapy in the world, Spark Therapeutics (NASDAQ: ONCE), which could become the first to win FDA approval of a gene therapy next year, and Bluebird Bio (NASDAQ: BLUE)—not to mention academic groups pursuing treatments for hemophilia and other diseases—has helped bring gene therapy back from the depths. A number of gene therapy companies have gone public over the past few years, and Celladon was one of them. It had the financial support of a number of corporate venture arms, like Pfizer Ventures and Novartis Venture Funds, became the first company to win a “breakthrough therapy” designation from the FDA for a gene therapy, and raised $44 million in an IPO in January 2014.

But despite the progress, there have been some setbacks in gene therapy as well, and Celladon unfortunately became one of the prime examples. Its gene therapy, Mydicar, a proposed treatment for patients with severe heart failure, failed miserably in a mid-stage study in April. Shares immediately plummeted 80 percent, and Celladon was forced to begin layoffs and search for strategic alternatives. Celladon’s shares had closed as high as $27.26 apiece in March, leading up to the data release. They’ve been worth just over $1 over the past several months during the trial fallout.

Those shares roughly doubled this morning following the announcement of the Eiger deal. Through it, a syndicate of investors new to Eiger—RA Capital Management, Sabby Management, Sphera Global Healthcare, Perceptive Advisors, and Monashee Capital Partners—-has teamed with longtime Eiger backers ViVo Capital and InterWest Partners to put $39.5 million into the new company. Eiger will have over $60 million in cash after the merger is completed, though the deal has to be approved by Celladon’s shareholders first. The cash should be enough to get Eiger through Phase 2 trials for at least two of its four programs by late 2016.

The company’s lead drug, lonafarnib (Sarasar), was originally developed by Schering Plough (now owned by Merck) and tested for a variety of cancers. Eiger licensed the drug from Merck, and is now developing it as a treatment for hepatitis D.

Celladon will issue about 85 million new shares to Eiger shareholders and the investors providing the financing. That’ll leave the new syndicate with 33 percent of the company, Eiger stockholders with 45 percent, and current Celladon equity holders with 22 percent.

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Slowing the Global Spread of Climate-Sensitive Infectious Diseases

Thu, 11/19/2015 - 08:20

Climate change is already suspected of causing legions of deaths every year as a result of extreme weather events: heat waves and floods; degradation of water supplies and sanitation with negative impacts on agriculture; and air pollution, which, in 2012, was responsible for 7 million deaths—one in eight deaths worldwide.

But climate change is also spurring an altered pattern of devastating infectious diseases on just about every continent today. Vector-borne diseases in particular—which are primarily spread by insect hosts and which cause more than one million deaths annually—are highly sensitive to alterations in the weather. The reason for this is that the behavior of the vectors themselves is sensitive to changes in the environment.

Indeed, vectors—living organisms, such as bloodsucking insects that can transmit infectious diseases between humans or from animals to humans—respond to a variety of climate variables, including temperature, precipitation, humidity, sea level, wind, and daylight duration. As our planet warms, the behavior of vectors is changing, and diseases like Chagas disease, dengue fever, leishmaniasis, malaria, chikungunya virus, and West Nile virus are migrating to northern climates, which have seen a pattern of temperature increases in recent years.

A good example is chikungunya virus, which is transmitted to humans by mosquitoes. Initially endemic in Asia and Africa, the disease has spread to the Caribbean, then gradually crawled north, and has now gained a toehold in Florida.

It’s also worth looking at Southern Asia, where malaria is already one of the most important vector-borne diseases. As changes in temperature and precipitation patterns take hold, malaria’s geographical range is expected to expand into more temperate and arid parts of the region. In India, for example, malaria distribution could well extend to higher latitudes and altitudes in the north, which have previously been unaffected by the disease. Due to the lack of tools for diagnosis, treatment, and prophylaxis for many of these diseases we are not prepared for this expansion pattern.

Right now, for instance, dengue fever, a viral infection spread by mosquitoes, mostly afflicts people below the equator—in South America, Africa, and South Asia. But researchers at the University of East Anglia say that the disease could move northward and break out in the highly populated coastal areas of the Mediterranean and Adriatic seas as well as the northeastern part of Italy, particularly the Po Valley.

A global research team led by the University of Pittsburgh Graduate School of Public Health has also recently shown that epidemics of dengue fever across Southeast Asia seem to be linked to the unusually high temperatures that accompany El Niño weather.

Moving from geography to medicine, the future is also worrisome. We’re making progress on a vaccine for dengue fever, but the first licensed vaccine is unlikely to protect completely against the disease. There are drugs to treat Chagas disease, but they are toxic, there is no approved vaccine, and diagnostics are crude to non-existent for different forms of this disease. We need more funding to combat chikungunya virus. And we’re working hard on a vaccine for West Nile virus.

Most of the global-disease-related headlines of the past decade have been devoted to the Ebola virus, SAARS, and MERS, which have been transmitted from human to human, thanks to a different effect of modern technology: rapid spread of isolated incidences through urbanization and global air transportation.

Now we should add the ways that climate change is changing the infectious disease landscape to our awareness and begin to ask how to address this significant global challenge.

We need to enhance vector surveillance and correlate it to climate information. Modeling and prediction of weather-related trends in vector movement and disease emergence could allow mapping in anticipation of countermeasure deployment.

Better science will also help us to understand the relationship between climate change and health outcomes. We need to study the linkages between climate and infectious disease transmission on a local or regional basis so that we can develop predictive models that are both relevant and actionable. Then we can develop and implement realistic preparedness and response plans for these health threats.

At the same time, we need to keep funding—and developing—cutting-edge, next-generation vaccines and diagnostics that will help us combat illness that is being accelerated by powerful and palpable global warming trends.

The changing burden of infectious diseases as a result of climate change could be quite onerous for the world. With the correct application of modern science, research anticipating these dynamic disease trends and the right application of available data we can help avert a burden of morbidity and mortality that could befall millions of people everywhere.

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“Innovation” a New Campaign Theme for Local Politics in San Diego

Wed, 11/18/2015 - 02:30

Something unusual has been happening to the innovation community in San Diego. Local pols are starting to pay more attention to entrepreneurship, and to the clusters of innovative tech and biotech startups sprouting throughout the region.

In contrast to San Francisco, where local politics gets polarized over tech startups like Airbnb and Uber, San Diego’s startup scene doesn’t usually provide much fodder for the body politic.

In San Diego, local elected officials, candidates, and voters are more likely to get into a political lather over a proposed real estate development—any development—or whether the San Diego Chargers are really, really, this-time-they-mean-it planning to leave town.

But something is different in this campaign cycle. A number of political candidates have been touting their support for that innovation thing that entrepreneurs do.

The list includes Barbara Bry, a tech entrepreneur and investor who is running for an open seat on the San Diego city council; Gil Cabrera, a candidate for city attorney; and Denise Gitsham, a Republican challenging the incumbent Democrat, U.S. Rep. Scott Peters in the 52nd Congressional District.

If elected, Bry told me, “I would be the first tech entrepreneur on the San Diego City Council.”

Whatever is happening, it’s not limited to political candidates. San Diego Mayor Kevin Falconer gave a Tedx talk on “fostering innovation,” and he was on hand when a new Fab Lab facility was dedicated in downtown San Diego. He speaks frequently about innovation, and made it easier for the organizers of San Diego Startup Week to use the Broadway Pier for their opening ceremony in June.

“I do feel that there is a bit of a sea change” that’s been positive for the tech community, says Austin Neudecker, an entrepreneur and mentor who has helped build the startup ecosystem in San Diego by advising companies, founding Startup San Diego, and organizing mentoring programs for startups admitted to the no-strings-attached EvoNexus incubator. “I don’t know if it’s just [politically] popular, or what.”

Of course, if you’re a political candidate, who is going to be opposed to innovation? It’s like motherhood and apple pie.

Tom Shepard

Tom Shepard, a longtime local Republican political consultant, says that with a few exceptions the leaders of San Diego’s innovation economy were not engaged in local politics in the past. “They were focused on the state and federal level, where they retain lobbyists to work on the issues that are important to them,” Shepard says.

But Shepard says that began to change in 2012. That’s when Qualcomm founder Irwin Jacobs crossed party lines to endorse Carl DeMaio, a conservative Republican, instead of Democrat Bob Filner, in the city’s mayoral campaign. Although Filner defeated DeMaio in the November general election, he resigned the following August amid allegations that he had forcibly kissed, groped, and sexually harassed at least 19 women.

A special election to fill the remainder of Filner’s term (which ends in 2016) led to city councilman Kevin Falconer’s inauguration as San Diego mayor in early 2014.

As Falconer settled into his new role, some tech entrepreneurs sought his support for making downtown San Diego more of a hub for tech startups. They enlisted the new mayor to help get Google Fiber to bring its gigabit Ethernet service to San Diego. They also asked Falconer and other city officials to consider turning the city’s old downtown library into a startup incubator for new tech companies—a proposal that City Council president Todd Gloria seized upon.

Neudecker told me he’s “extra happy” that Gloria has been vying with Falconer for props as a local tech booster, saying, “I would rather them fight over which one of them is doing a better job of supporting the tech community.”

According to Shepard, there’s now an evolving sense that San Diego’s identity is tied to the early stage companies advancing innovation in technology and the life sciences.

“Those companies are a driving force in growing our local economy,” Shepard says. Fast-growing companies contribute directly to the local economy, and they pay better-than-average salaries to attract scientists and other highly skilled workers.

Many of those companies—and their employees—are clustered among San Diego’s coastal communities encompassed by the 52nd Congressional District, which Peters claimed in a narrow victory over Republican Brian Bilbray in 2012.

Gitsham, a Republican who announced her candidacy earlier this month, moved to San Diego in 2010 while working for Sapphire Energy, the San Diego industrial biotech, and started a small public relations firm, now called SVN Public Relations in 2012.

Her firm helped promote San Diego’s Startup Week in 2014, and Gitsham said she particularly wants to speak up for the district’s creative, technology-based start-ups and entrepreneurs.

But incumbency has its privileges.

Last week, Biocom, the San Diego life sciences industry group, gave Peters its “Elected Official of the Year” award. The group cited Peters for his advocacy on behalf of federally funded scientific research, for co-founding a “life science caucus” in the House, for working to repeal a federal tax on medical devices, and for advocating for balanced and targeted patent reform.

“The life sciences have come to the vanguard of the innovation economy in San Diego,” Biocom CEO Joe Panetta says. “It’s significant from a political standpoint because biotech is creating jobs, jobs that are paying very well. And it’s a segment of the population that votes.”

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Keeping Score, Clinical Watchdogs Push Drugmakers For More Open Data

Tue, 11/17/2015 - 08:30

Should drug companies publicly release all data from the tests they conduct on human volunteers? A growing number of parties say yes, and last week, a group of academics unveiled a scorecard to spotlight companies that have recently kept their clinical data hidden from public view—and those that have made the data available.

As with all shorthand systems that boil down complicated scenarios, the clinical trial scorecard, developed at New York University, Harvard University, and Yale University, requires explanation.

I’ll get to that. But what struck me like a bolt was the organizing principle of the report’s authors. It was simple, perhaps even unassailable: In exchange for the privilege of testing for-profit drugs on human subjects, companies have an obligation to make all their clinical data available for the common good. Science and medicine should work best when research can be examined, criticized, and serve as a foundation for more studies.

But when drug companies hold back results, positive or negative, there is no common foundation to build upon. “How can we practice evidence-based medicine if we don’t have all the evidence?” said Jennifer Miller, the report’s lead author and an assistant professor in the division of medical ethics at New York University School of Medicine.

Miller, who said she has been working on the project for six years, expects her scorecard to become an annual tally, but last week’s release in the journal BMJ Open started with a small sample size. She and her team assessed 318 trials used to test 15 drugs, all approved by the FDA in 2012. This is a subset of all new drugs approved that year; the authors decided to investigate only drugs brought to market by large companies. With their size and financial muscle to put behind the transparency requirements, “if anyone would score well, it would be [them],” said Miller.

Not all scored well. Before I get to the names, it’s important to describe the scorecard. It has two parts. The first part measures how companies complied with FDA regulations, implemented in 2007, which require some disclosure of data that leads to a drug approval. But those regulations are a low bar, said Miller—“a minimal set of standards” that only require a fraction of the body of evidence to be disclosed.

Instead, what Miller and colleagues see as the gold standard of clinical disclosure is the second part of the scorecard, which tallies the percentage of trial results that are either reported in summary or published in full—a much broader set of data than required by FDA. In other words, by emphasizing this part of the scorecard, Miller and colleagues want drug companies to follow the spirit of the idea of data for the common good, not just the narrow letter of the 2007 law. That said, this is not just an academic idea: Miller noted that the FDA’s parent department, Health and Human Services, has ethical language in its “common rule,” which in turn was based on a 1979 report on the ethics of research with human subjects.

Some companies fared quite well. Under the scorecard’s ethical gold standard, three companies published all trials—perfect scores—that led to their 2012 drug approvals: Pfizer, for the drug axitinib (Inlyta, approved for renal cell carcinoma); the Janssen division of Johnson & Johnson for bedaquiline (Sirturo, for tuberculosis), and GlaxoSmithKline, for an infants’ vaccine branded MenHibrix.

At the bottom of the list, the three worst scores belong to Gilead Sciences, for its work on the HIV four-drug combination sold as Stribild (only 21 percent of trial results are publicly available); Sanofi, for the multiple sclerosis drug alemtuzumab (Aubagio, only 22 percent of results available); Sanofi again, for the colorectal cancer drug ziv-aflibercept (Zaltrap, 40 percent); and Pfizer and its development partner Protalix, for the Gaucher disease treatment, taliglucerase alfa (Elelyso, also 40 percent).

A Gilead spokesman said the company had no comment. A Sanofi spokeswoman responded that the company looks forward to reviewing the study, and that it complies with the drug industry’s own guidelines for sharing clinical trial data. And a Pfizer spokesman said Elelyso data disclosure was the responsibility of Protalix.

The Pfizer spokesman also noted the high marks Pfizer received not just for axitinib but for trials related to two other drugs approved in 2012. He said Pfizer has taken multiple steps in what he called “a longstanding commitment to clinical transparency.” For example, Pfizer is working on several data-sharing projects and collaborative clinical trials.

Bayer, which made public only 42 percent of trial information related to its colorectal cancer drug regorafenib (Stivarga), sent a statement via a spokeswoman that said the company makes data from its trials available on its website and on the trial registry “regardless of the outcome of the study.”

Nearly every company pays lip service to a commitment to transparency. (Search on a company’s name and “clinical data transparency” to find their webpages.)

But which ones have gone beyond lip service? Miller went out of her way to praise Pfizer and GSK. “They’ve cleaned up their act,” she said. “They’ve gone above and beyond the legal requirements.”

For GSK, that “act” once included the cover-up of negative data around the antidepressant paroxetine (Paxil). It was one of GSK’s predecessor companies, SmithKline Beecham, which had paroxetine approved for adults in 1992 and continued to study it as a potential treatment for teenagers.

That study, dubbed Study 329, was originally positive and “influential in the literature supporting the use of antidepressants in adolescents,” wrote researchers who revisited the notorious study—long the target of retraction efforts … Next Page »

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Stealth Co Unveiling, New Speakers Added to Nov. 17 Healthcare Event

Fri, 11/13/2015 - 20:29

Two great new speakers—one of whom will bring a major new company out of stealth mode—have just joined the lineup for Tuesday’s Healthcare Summit 2015. If you were on the fence before, you should get off it fast. If you are interested in the future of healthcare, you will not want to miss these new elements.

The summit takes place at the Broad Institute in Kendall Square on Tuesday afternoon, Nov. 17. You can find out more and get your tickets on our event site.

The new speaker we can name is Kathy McGroddy-Goetz of IBM Watson Health. She will be talking about what she calls the emerging era of cognitive computing. The first computing era, she says, was when computers were basically used as tabulating systems—counting things like the U.S. census. Then the field migrated into programmable computers: think ‘if–then–else.’ And now (and this is where Watson Health comes in) we have the cognitive era, where computers can power through vast amounts of structured and unstructured data, use context, and learn—and humans can interact with them and utilize their findings in a much more natural way.

McGroddy-Goetz says she plans to bring the power of the cognitive era home for the audience with specific examples of “patient journeys” that show what Watson is enabling right now, as well as what is on the horizon.

Our other new speaker is He-Who-Cannot-Be-Named, but only for the best of reasons: his identity is a surprise. What we can say is that he will be giving us the early, first-hand word about a major new company backed by major venture investors from Boston and beyond and coming out of stealth the morning of our event.

These two will be joining an all-star cast. Kicking off the afternoon will be a chat between scientist-entrepreneur Tillman Gerngross of Dartmouth University and Terry McGuire of Polaris Partners. Other speakers include Lynda Chin, chief innovation officer for health affairs for the University of Texas system; MIT biologist Leonard Guarente, a specialist on health and aging and co-founder of Elysium Health; Sheila Dodge from the Broad Institute; and Boston Children’s Hospital chief innovation officer John Brownstein.

The afternoon’s program will cover the hottest life science topics, from how diagnostics and data are reshaping healthcare to new models for scientific and strategic collaboration. In the center of it all, executives from three hot health IT startups—PillPack, Wellframe, and CoPatient—will explain their plans to put healthcare into consumers’ hands.

Again, you can view the agenda and get your tickets here. We promise, you will not want to miss this event. We hope to see you next Tuesday afternoon, Nov. 17.

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Advancing Drugs at Roche, Sophiris, Coherus, and Other Bio News

Fri, 11/13/2015 - 16:53

An economic impact report released this week by the California Life Sciences Association found that life sciences companies in the Golden State currently have 1,235 new drugs in the development pipeline—and it seemed like this week there was news on each one. Here’s our wrap of the life sciences news from the West Coast

—The FDA approved cobimetinib (Cotellic), which was first developed by South San Francisco, CA-based Exelixis (NASDAQ: [[ticker: EXEL]]) before Genentech, now part of Roche, bought much of the rights and took it through late-stage clinical trials for advanced melanoma. It was approved as a combination therapy with Genentech’s vemurafinib (Zelboraf). The expected approval is a big step back for Exelixis, as we wrote about in August. The biotech will split cobimetinib profits with Genentech.

—In a reversal of fortune, San Diego-based Sophiris Bio (NASDAQ: SPHS said topsalysin, its biologic drug for treating an enlarged prostate, met the primary goal of a 52-week clinical trial by showing a significant improvement in patient symptoms that include frequent or difficult urination. The results sent shares of Sophiris soaring—roughly 11 months after the stock tanked after Sophiris reported interim results (three months into the trial) that failed to show effectiveness.

Amplyx Pharmaceuticals said it closed on $40.5 million in Series B financing, and plans to use the capital to advance development of APX001, a new anti-fungal compound for treating life-threatening fungal infections. RiverVest Venture Partners led the round, which included NEA, BioMed Ventures, and individual investors. Amplyx intends to take advantage of incentives created as part of the GAIN Act of 2012.

—One of the rare small or modestly sized firms working on biosimilars hit a clinical milestone, too. Coherus BioSciences (NASDAQ: CHRS) of Redwood City, CA, and its development partner Baxalta (NYSE: BXLT) announced positive Phase 3 data for their biosimilar based on etanercept (Enbrel). It remains to be seen, however, if the wave of biosimilars heading toward market will make a noticeable dent in the staggering cost of U.S. healthcare.

—Amgen is also working on biosimilars, and its copycat version of adalimumab (Humira), the world’s top-selling drug, posted good Phase 3 results this week.

—San Diego’s Avalon Ventures formed Iron Horse Therapeutics, a new biotech with a promising new drug candidate for treating amyotrophic lateral sclerosis (ALS), a neuro-degenerative disease also known as … Next Page »

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How Foreign-Born Workers Spark Employment and Innovation

Thu, 11/12/2015 - 18:49

The way critics tell it—including presidential hopefuls as varied as Bernie Sanders and Donald Trump—high-skilled foreign workers are job stealers. If these candidates have their way, living and working in the U.S. will become harder, not easier, for foreign talent.

This protectionism, the theory goes, will positively impact the quantity and quality of American jobs: one, by sheltering the American worker from foreign competitors, and two, by preventing American companies from hiring talented foreigners willing to work for less.

The truth is foreign talent has not been shown to suppress wages and has in fact grown American employment and innovation. Forty percent of Fortune 500 companies are run by foreigners or their offspring, including Yahoo!, Google, Intel, and YouTube. Educated immigrants secure patents at around double the rate of educated, native-born Americans. Cities that have more foreign professionals employed in high-skill, specialty occupations have been shown to have lower unemployment rates and more robust entrepreneurial environments. In other words, since these entrepreneurs are starting and growing a new business, there is no job for them to steal. Instead, this immigrant creates American jobs and is helping to stimulate and drive our economy.

Immigrants are almost twice as likely to start businesses than native-born Americans, according to research by the Ewing Marion Kauffman Foundation. And despite far lower employment numbers overall, immigrants were 27 percent of all U.S. entrepreneurs in 2012, up from 14 percent in 1996. In Silicon Valley, immigrant founders started 52 percent of companies between 1995 and 2002, a period that has spawned unprecedented growth and productivity. Engineering and technology companies started by immigrant founders in 2012 employed 560,000 workers and produced $63 billion in sales. There would, in a word, be no Silicon Valley without immigrants. This is why business leaders like Mark Zuckerberg and Bill Gates support immigration policy reform that will make it easier for talented entrepreneurs to start innovative companies, which will in turn grow America’s workforce, create American jobs, and bolster our competitiveness.

But the solution is not to limit the number of high-skilled foreign workers. A report in April by the Information Technology and Innovation Foundation found that U.S. universities are graduating only half the workers to fill the 150,000 computer jobs that open up each year. We desperately need these high-skilled foreign immigrants.

Rather, we must revise existing rules by, for example, giving the Department of Labor more leverage to punish rule breakers; and by establishing exacting displacement rules, which will require employers to prove that an American worker of comparable skills was not displaced to make room for a foreign worker. The 2013 Senate bill on immigration had 22 provisions to prevent misuse of current immigration policy.

We must also adopt new and fresh policies that will help us maintain our edge in innovation. The proposed Startup Visa, which some are calling the “Entrepreneur Visa,” has strong bipartisan support and backing from tech and business leaders, and is a timely and powerful step in that direction. The visa is intended to attract tech entrepreneurs and will require applicants to prove they offer an innovative product or service, or that they’ve received significant funding from U.S. investors. Priority will be given to those who can demonstrate that they are job creators, affiliated with U.S. accelerators and universities, and who earn advanced degrees in science and math from American universities.

In my 30 years in economic development, in which I’ve worked with roughly 700 communities nationwide, I have found that entrepreneurship and diverse populations go hand in hand—that communities that welcome foreign talent and innovation put themselves at a competitive advantage. The same is true at the country level. The island-sate of Singapore, void of natural resources, has become an economic powerhouse in no small part due to its embrace of foreign skilled-talent. Other countries, from Canada to Italy, are making the connection and revising their immigration policies accordingly.

It simply makes no sense that the United States currently has no immigration pathway for entrepreneurs. These are individuals who pose no threat to the American workforce. Rather, entrepreneurs bring jobs, innovations, and economic growth to our communities.

America is still the world’s leader in innovation and entrepreneurship. Keeping it that way will depend on putting our politics aside on this wedge issue long enough to enact bold, sweeping job-centric immigration reform.

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SkySpecs Takes Flight with $3 Million Series A, UpWind Partnership

Thu, 11/12/2015 - 15:41

SkySpecs, the Ann Arbor-based drone startup, announced this week that it raised $3 million in a Series A round. Venture Investors led the investment, with participation from Huron River Ventures, Amherst Fund II, the Michigan Angel Fund, and Invest Michigan. To date, the company has raised a total of $4.3 million, said SkySpecs CEO Danny Ellis.

“We are excited,” Ellis said. “Our team is looking forward to getting the technology in the field—that’s pretty exciting after working on it for so long in the lab.”

The investment caps a busy, high-growth year for the startup. One year ago this month, SkySpecs was the $500,000 grand-prize winner at the Accelerate Michigan Innovation Competition. Two weeks before winning Accelerate Michigan, SkySpecs headed east for a four-month stint in the Techstars New York accelerator.

“The mentorship at Techstars was incredible,” Ellis added. “Being plugged into their network is incredible. We were a team of engineers, so we didn’t have experience with business strategy or building a brand.”

Ellis said SkySpecs’ patent-pending collision-avoidance technology has the ability to “lock-on” and maintain a specified distance from a target object, increasing safety and reducing the need for costly human operator training. SkySpecs is working directly with commercial customers to launch its automated drone inspection service in early 2016.

Founded in 2012, SkySpecs initially aimed to make autonomous drones easy to use for non-pilots. “We made the software generic enough to work on any drone and added sensors so the drones could see the outside world,” Ellis said. “Then we realized collision avoidance was where we could make a real impact. We made the decision to focus on inspection and operate as a drone-as-a-service company.”

That decision also helped investors warm to SkySpecs. Federal regulators still haven’t established the rules that will govern commercial drone operators, and that uncertainty contributed to the reluctance of early backers, Ellis said. As of now, SkySpecs has been granted an exemption from the ban on drones, which means the company is cleared to fly commercially if it abides by the FAA’s strict interim rules.

“It’s very exciting because it means there’s a light at the end of the tunnel,” Ellis said. “Three years ago, there was no light. Investors were very shy about backing an essentially illegal business in the U.S.”

SkySpecs has also entered into a partnership with San Diego’s UpWind Solutions, a provider of operations and maintenance services for utility-scale wind-energy projects. UpWind uses SkySpecs’ collision-avoidance technology as it inspects and maintains wind turbines. Ellis said his company wants to perfect its software and hardware technology in the wind sector before it scales to other potential markets such as cell towers, mines, power transmission towers, and bridges.

SkySpecs, which has an office in downtown Ann Arbor, plans to use the money from the Series A to expand overseas and double the current team of eight employees as it waits for the FAA to finalize commercial drone regulations.

“The ruling on small drones is supposed to happen in June,” Ellis said. “It should really clarify what people can do commercially with drones that weigh less than 55 pounds. Then, after that, I think we’ll see the market explode.”

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Amplyx Raises $40.5M to Advance Drug for Deadly Fungal Infections

Wed, 11/11/2015 - 05:02

Sometime after the Irish drug giant Shire acquired San Diego-based Lumena Pharmaceuticals last year, Lumena CEO Mike Grey went back to work on a new startup—but he wouldn’t say too much about what he was working on.

He is now. Grey and a couple of other key executives from Lumena have joined Amplyx Pharmaceuticals, one of those frugal drug discovery companies that seem to thrive in San Diego. But Amplyx has taken a recent turn that is taking the little company to a whole new level.

Today Amplyx says it has closed on $40.5 million in Series B financing, and plans to use the capital to advance development of a new anti-fungal compound for treating life-threatening fungal infections. St. Louis, MO-based RiverVest Venture Partners led the round, which included investments by New Enterprise Associates, BioMed Ventures (the venture arm of San Diego’s BioMed Realty Trust), and individual investors.

Amplyx was founded in 2006, and has managed to continue its small-molecule R&D, which is focused on finding ways to improve existing cancer and anti-viral drugs, by securing angel funding and at least $7.7 million in NIH Small Business Innovation Research grants.

Amplyx also was among the first 18 startups to get low-cost lab space at San Diego’s Janssen Labs, the “no strings attached” innovation center operating under the corporate umbrella of Johnson & Johnson.

Grey said yesterday he began helping Amplyx co-founder and CSO Mitchell Mutz about a year ago as an informal adviser. Amplyx later in-licensed a promising drug compound for treating fungal infections from an undisclosed pharmaceutical company.

It was an unusual move, Grey said, and outside the company’s usual focus on improving existing drugs. For one thing, the compound Amplyx calls APX001 works in a new way and represents a new class of anti-fungal drugs—and the FDA hasn’t approved a new class of anti-fungal agents since 2001.

Grey joined Amplyx as CEO, along with Ciara Kennedy, who led the work on cholestatic liver disease at Shire and Lumena (and is now COO at Amplyx), and Susan Dube, who was Lumena’s vice president of corporate development (and will head business development and administration for Amplyx).

“Amplyx was a research enterprise, and they’re doing good work,” Grey said. “But they needed some [drug] development expertise.”

With the venture funding announced today, Grey said he expects Amplyx to grow from its current headcount of 14 employees to 20 or more in the next six months.

By next year, Amplyx plans to initiate an early stage trial of APX001. The company says it is developing both intravenous and oral versions of the drug, and plans to evaluate the safety and tolerability of both formulations.

From there, Amplyx plans to develop a series of mid-stage trial programs for testing APX001 against candidiasis, invasive aspergillosis, and certain rare molds.

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In Significant Reversal, Prostate Drug Met Primary Goal After All

Tue, 11/10/2015 - 17:03

After getting something worse than a lump of coal for Christmas last year, Sophiris Bio got an early present for the 2015 holidays— its experimental prostrate drug met the primary goal of a late-stage study. Shares of Sophiris Bio (NASDAQ: SPHS) more than quadrupled overnight—opening today at $3.28 from yesterday’s close of 86 cents a share.

Last December, shares of Sophiris, based in San Diego and Vancouver, BC, plunged more than 80 percent after the company reported interim results, saying the experimental drug failed to meet a pre-defined threshold of efficacy after three months.

But that was then.

In final results following the 52-week pivotal trial, Sophiris says its drug candidate topsalysin demonstrated a statistically significant improvement among men experiencing lower urinary tract discomfort following a single treatment with the drug. Sophiris has been developing topsalysin as a therapy for treating an enlarged prostate, also known as benign prostatic hyperplasia, (BPH).

Topsalysin is a genetically altered form of the naturally occurring protein proaerolysin. Sophiris says BPH affects most men in their 60s. As the prostate enlarges, the walnut-size gland gradually press against the urethra, leading to an unhappy condition that requires frequent urination or difficult urination. Standard drugs or surgery can lead to erectile dysfunction or cardiovascular side effects.

In a statement today, Sophiris says “the drug continues to demonstrate a favorable safety profile, with no evidence of any treatment related sexual or cardiovascular side effects.”

Sophiris shares settled after the opening, and were hovering above $2.50 in mid-day trading.

How did things turn around so dramatically for Sophiris?

“The thing that’s important to understand about the interim analysis is that it wasn’t a true interim analysis,” Sophiris CEO Randall Woods told me by phone this morning. Rather, the interim results were intended to serve as a snapshot of how well the clinical trial was meeting a pre-defined efficacy threshold after three months, as determined by an independent data monitoring committee.

If the trial met the efficacy threshold at three months, Sophiris intended to proceed with laying the groundwork for another late-stage trial, Wood said. In other words, the interim results “really were for our own internal planning purposes.”

With the positive final outcome reported today, Woods said Sophiris would begin planning for that late-stage trial, which would be needed for the company to seek regulatory approval of the drug. To complete the trial, Sophiris would need to raise additional capital or find a big pharma partner, Woods said.

Sophiris COO Allison Hulme, who oversees research and development, says a final analysis found that patients who received the drug began showing improvement in their urinary discomfort in week eight, but the maximal effect was around the 18th week of the study. “The analysis showed us a good [almost] 8 point improvement [using the International Prostate Symptom Score] that was maintained through the rest of the study,” Hulme said.

Sophiris has genetically modified proaerolysin so that it can be activated by only one enzyme—the Prostate Specific Antigen (PSA), which is found only on the surface of prostate cells and nowhere else in the body.

The drug is deposited by an ultrasound-guided injection in the area where the prostate meets the ureter. Once activated by PSA, the protein drills into the prostate’s cell walls, triggering a programmed cell death known as apoptosis—a process that shrinks the prostate without affecting the ureter or surrounding tissues.

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Milestones of Innovation 11: Blackouts and the Safety of the Grid

Mon, 11/09/2015 - 05:01

While we party upstairs in our micro-age of whirling electrons and dancing apps, there’s some rumbling down in the basement. In a new book called Lights Out, the television journalist Ted Koppel has been reminding us to be very worried about the reliability of one of the central innovations of modernity. He’s talking about our little-understood electricity grids, including the one that spreads from Ontario to Florida, and from Maine out to the middle of Nebraska.

Koppel’s book, focusing on the dangers of an attack on the grid by cyber-hackers—he argues that we have no real plan to prevent it or recover from it—is timely. Fifty years ago, on Tuesday, Nov. 9, 1965, the American northeast learned frighteningly how much we depend on something very macro: the silently evolving electricity grids that have become more and more important to us since we began to depend on them in 1890s.

Just as people were heading home from work on a cool, mercifully moonlit evening, switches that had been set wrong near Niagara Falls operated inappropriately and sent power surging the wrong way in the network of power lines that blanketed Ontario, New York, and New England. Twenty-three million customers were plunged into darkness that lasted hours.

It was scary. Two hundred airliners headed toward New York’s airports suddenly didn’t have runway lights to assist their landing, and had to be re-routed. Hospitals began struggling to mobilize emergency generators to keep urgent surgery going. Traffic lights went dark and citizens turned into traffic cops. People were trapped in elevators, and many more couldn’t get down from their skyscraper apartments. With city subways halted, many thousands had to trudge miles homeward.

Electric utilities worked through the night to turn the lights back on, painstakingly reconnecting interrupted systems, while an entire region coped, relatively cheerfully. But it was also clear that the system needed much more provision for emergency power, and a lot better understanding of how to control malfunctions. It needed more of a brain and more centralized control.

Massive fixes followed, including regional control centers like the ones in Holyoke, MA, and Guilderland, NY. supervised by a partly voluntary national electricity reliability authority in Princeton, NJ.

But the responses didn’t completely solve reliability or recovery problems of a fantastically complex system. As in other parts of the world, the big northeastern grid was challenged again.

Because of multiple lightning strikes on a sultry summer night, New York City suffered a city-wide blackout on Wednesday, July 13, 1977. Luckily, the blackout was limited to the nine million people served by a single utility, Consolidated Edison, but massive looting broke out in many neighborhoods. Furthermore, the system had undergone some changes since 1965 that actually made it harder to recover. The blackout actually took twice as long to end as the events a dozen years before.

And in 2003, a power line sagging into an untrimmed tree in Ohio set off a chain of failures that plunged 50 million people in the American northeast into darkness on Thursday, Aug. 14, 2003. Enough was enough. This time, a federal law gave the Princeton reliability agency full authority to set rules.

Power grids arise from the nature of that mysterious “fluid,” electricity. Except in batteries, electricity is difficult to store. In general the output of a spinning generator has to be matched by power demand. And that demand is varying, second by second, minute by minute, hour by hour, in a particular location. It goes up when people wake and get ready to go to work or school, and again in the afternoon and evening when people head home, prepare dinner, and do the laundry. It goes down during the day and even more at night. It swells on hot afternoons when people turn on air conditioners.

Those generators cost huge amounts of capital, creating intense pressures to economize. A remedy is to share power over wide regions. Such sharing sprang up in both Germany and America soon after Thomas Edison’s package of innovations in 1882 that created a practical system to light homes and offices more brightly and cleanly than gas lamps.

If something causes a surge in the system, that fault must be isolated. But, as in 1977, a system that got rid of sulfur-spewing local coal-fired generators becomes more reliant on electricity from long-distance power lines. And lightning can hit transmission lines four times in an hour—after the crews of the back-up gas-fired generators on barges have gone home. Engineers can calculate the wrong settings for a circuit-breaker. Or a system controller can remain ignorant that he doesn’t have a particular power line open, because the display for it is in the next room—so he hesitates to “shed load” by cutting off part of the city. Or the massive underground electricity supply lines for a city can lose power for the generators that circulate their refrigerated oil coolant—so it takes hours to re-energize them.

Our Lady of Disasters keeps finding new points of vulnerability in the fabric of modern life. As we back off storing more and more greenhouse gases in our stratosphere, and move to “smarter,” more decentralized electricity grids with swiftly-varying power supplies from solar panels and windmills, humanity will face even sterner tests of the reliability of our basic systems.

[Editor’s Note: This is the eleventh of a series of notes about major anniversaries in innovation and what they teach us. You’re invited to suggest other milestones of innovation for the Xconomy Forum. Example: early in 2016 is the 200th anniversary of the miner’s safety lamp.]

Further reading:

Ted Koppel, Lights Out: A Cyberattack; A Nation Unprepared; Surviving the Aftermath, New York, Crown Publishers, 2015; lead item, “CBS Sunday Morning,” Nov. 1, 2015.

Victor K. McElheny, “Improbable Strikes by Lightning Tripped Its System, Con Ed Says,” New York Times, July 15, 1977, A2.

Victor K. McElheny, “Technology: The Blackout Load on Phone System,” New York Times, July 20, 1977, D1 and D13.

Victor K. McElheny, “Con Ed President Says System Designed to Avert Blackout Actually Was a Factor in Bringing It On,” New York Times, July 21, 1977, 28

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Avalon, GSK Extend Collaboration with Startup Focused on ALS

Mon, 11/09/2015 - 05:00

[Corrected 11/12/15, 9:30 am. See below.] San Diego’s Avalon Ventures has started a new biotech focused on Lou Gehrig’s disease, the neuro-degenerative disease that claimed the life of the Hall of Fame New York Yankees first baseman, whose endurance gave rise to his nickname as “the Iron Horse.”

With the launch of the aptly named Iron Horse Therapeutics, the seventh biotech founded under Avalon’s collaboration with GlaxoSmithKline, the venture firm says it also is extending its partnership with GSK, after tweaking the terms to increase its potential payout by 20 percent.

“The collaboration we cooked up two-and-a half to three years ago is doing so well, we wanted to expand it,” Avalon partner Jay Lichter said. Under their “build-to-buy” collaboration, Avalon and GSK initially agreed to create and fund 10 biopharma startups through the development of clinical candidates, at which point GSK holds the first option to acquire the startup.

The collaboration, established in April 2013, combines GSK’s resources and R&D expertise with Avalon’s knack for choosing a particular life sciences innovation and starting a new company around the technology. So far, the Avalon-GSK collaboration has resulted in the creation of six biopharma startups in San Diego: Sitari Pharma, Silarus Therapeutics, Thyritope Biosciences, Adrenergics, Cadherx Therapeutics, and Calporta Therapeutics.

None of the six startups have reached an exit yet, so the overall return-on-investment of the Avalon-GSK portfolio remains unknown. But the concept of combining the nimble startup mentality of a VC firm with the scale of big pharma has been serving as a new business model for venture-backed innovation, and Avalon’s Lichter said he’s had numerous conversations with other pharmaceuticals about the collaboration.

Avalon originally pledged $30 million from its tenth fund to the effort ($3 million up front per startup), while GSK agreed to invest $7 million for each startup. Altogether, GSK committed as much as $465 million for seed funding, R&D support, and milestone payments.

Under their revised agreement, Lichter said there is no limit on the number of startups. Avalon and GSK will continue indefinitely to jointly invest $10 million in Series A funding for each new company. Each one will take root at COI Pharmaceuticals, a shared “community of innovation” that Avalon has established in San Diego as a fully equipped R&D facility. Avalon also provides operational support and executive leadership, at least until each startup can validate its technology enough to begin operating more independently.

[Corrects amount of earn-outs] The revisions, though, enable Avalon to earn as much as $50 million in potential milestone payments for each company that brings a new drug to market. Avalon’s potential milestone earn-outs were $40 million under the original terms, Lichter said.

The $10 million joint investment that Avalon and GSK are making in Iron Horse Therapeutics “closes … Next Page »

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West Coast Biotech Roundup: KaloBios, Atreca, Acadia, Twist & More

Fri, 11/06/2015 - 17:02

There were drips and drabs of news out west this week, but compared to recent weeks with former Silicon Valley darling Theranos taking a beating, or 23andMe revving its engines, it’s been sleepy. The big exceptions come in the form of two Bay Area companies going in opposite directions. The big green blinking arrow pointing up, if that’s your emoji of choice for what’s hot, goes to Stanford University spinout Atreca, which said it had rounded up $56 million for a Series A. The pulsing red arrow pointing down is for KaloBios, an antibody drug maker that hasn’t had good news in a long time.

Let’s find out more about those two companies, plus more news from the Pacific side of the country, where you never know what you might see as you finish up a tour of Alcatraz.

—KaloBios Pharmaceuticals of South San Francisco, CA, continues to spiral downward, announcing Thursday it would lay off 17 people, more than half its employees, and continue to look for a buyer. It will also halt a clinical trial of a blood cancer drug and likely delay its next quarterly earnings report. Five years ago, then-CEO David Pritchard talked about building a billion-dollar company. Instead, KaloBios is limping badly. Pritchard left the company abruptly in January.

—Atreca of Redwood City, CA, said it has pushed its Series A financing all the way to $56 million, a figure that includes at least $6 million invested by the Bill and Melinda Gates Foundation in 2012. As Xconomy reported at the time, Atreca spun out of Stanford University with technology that surveyed the entirety of various people’s immune responses, potentially to find better antibody drugs or vaccines to fight cancer or infectious disease. The lead investor in the round is an undisclosed U.S. healthcare-focused fund.

—San Diego’s Acadia Pharmaceuticals (NASDAQ: ACAD) said Monday that the FDA has set a May 1 target date to consider Acadia’s new drug application for pimavanserin (Nuplazid) as a treatment for Parkinson’s disease-associate psychosis. The date was set as a priority review, which the FDA applies to drugs that might offer major advances in treatment or treatment where no adequate therapy exists.

—San Diego’s Regulus Therapeutics (NASDAQ: RGLS) said it has expanded development of its experimental drug candidate RG-101 to treat hepatitis C under a collaboration with GlaxoSmithKline (NYSE: GSK).

– Synthetic DNA maker Twist Biosciences of San Francisco closed a deal with Ginkgo Bioworks of Boston to sell Ginkgo 100 million base pairs of synthetic DNA, which the companies claim is 10 percent of the total capacity worldwide. Ginkgo said it would use the raw material to design organisms that help produce new fragrances, foods, and industrial products.

—Shares of San Diego’s Kura Oncology (NASDAQ: KURA) declined slightly Thursday, its first day of trading on the Nasdaq exchange. The company, which is developing new drugs for solid tumors and blood cancers, priced its IPO Wednesday evening—selling 6.25 million shares at $8 a share to raise a total of $50 million, well below its original plan.

—Fresh off its October IPO, antibody drug developer CytomX Therapeutics (NASDAQ: CTMX) of South San Francisco, CA, said today it would collaborate with MD Anderson Cancer Center of Houston to create new cell therapies that use CytomX’s antibody technology to hone in more precisely on cancer targets.

—Juno Therapeutics (NASDAQ: JUNO) signaled confidence that its experimental T cell cancer treatments would continue progress toward marketing approval by hiring a chief commercial officer, Bob Azelby. Azelby was previously in charge of Amgen’s oncology business.

—VistaGen Therapeutics of South San Francisco, CA, has dosed the first patient in its Phase 2a trial of its lead compound, AV-101, to treat depression.

—UC San Diego said a $5 million gift from ResMed (NYSE: RMD) founder and chairman Peter Farrell will support sleep medicine research and care at the UC San Diego School of Medicine. San Diego-based ResMed develops medical equipment and devices for sleep-disordered breathing and related respiratory disorders.

—Medical practice software maker Quality Systems (NASDAQ: QSII) of Irvine, CA, agreed to buy San Diego-based HealthFusion Holdings for at least $165 million. HealthFusion develops Web-based software for physicians, hospitals, and medical billing services. Contingent payments could eventually add as much as $25 million to the deal.

—The San Diego Blood Bank announced a pilot program for volunteers to participate in genomics research as part of the federal government’s Precision Medicine Initiative. The blood bank says 100 volunteers already have agreed to donate an extra vial of blood to be sequenced for the nationwide genomics research study.

Xconomy San Diego editor Bruce V. Bigelow contributed to this report.

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Seven Major Cancer Centers To Pool Patients’ Gene, Health Data

Fri, 11/06/2015 - 14:30

Here and there, the genetic information of a cancer patient has helped a doctor find that person the right drug or steer her away from the wrong one. But the use of genetic knowledge to improve people’s health is in its infancy.

That’s why seven major cancer treatment centers in North America and Europe are pooling their patients’ data in a central repository, dubbed Project GENIE (Genomics, Evidence, Neoplasia, Information, Exchange), for their own researchers and doctors to use and, eventually, to open completely to all comers. “Right now genetic testing is getting done for individual patients and being returned to those patients’ physicians,” says Victor Velculescu, co-director of cancer biology and a professor at Johns Hopkins University who has helped push forward the field of cancer genomic analysis. “Those data are not being used, except to help that patient. This effort brings all data from all reports together and allows us to keep learning.”

The bet with this project—and writ larger, with the promise of so-called big data in all manner of medical fields, not just cancer—is that the more peoples’ information is pooled for comparison and analysis, the deeper the insights into health.

“It’s reasonable to think that it could have the records of 50,000 to 100,000 patients in several years,” says Charles Sawyers, one of the nation’s top cancer researchers and the chair of the human oncology and pathogenesis program at New York’s Memorial Sloan Kettering Cancer Center. “That’s a lot.”

Sawyers hinted earlier this year to Xconomy that the massive data sharing project was underway and could launch in the fall.

Sloan Kettering, Johns Hopkins’s Kimmel Cancer Center in Baltimore, Dana-Farber Cancer Institute in Boston, Princess Margaret Cancer Centre in Toronto, Vanderbilt-Ingram Cancer Center in Nashville, TN, Institut Gustave Roussy in France, and the Center for Personalized Cancer Treatment in the Netherlands are the seven participating institutions. Velculescu says the consortium could welcome more in due time.

One of the first projects GENIE could tackle is what Dana-Farber chief scientific officer Barrett Rollins calls a “meta” problem. Does decoding the genes of patients’ cancer actually lead to better health outcomes? Dana-Farber and other institutions have their anecdotal success stories; Farber has a handful of what it calls “Lazarus cases,” such as a young man with a seemingly intractable form of leukemia, who was at death’s door when a sequence of his cancer pointed toward treatment with the drug imatinib (Gleevec), according to Rollins. “Now he and his wife are having a baby,” says Rollins.

But no one has examined whether more sequencing, across large populations, is worthwhile. “This is one of our best hopes to demonstrate what we all intuitively feel is true,” says Rollins, noting that insurance companies aren’t keen on paying for tests that don’t have a body of positive health outcomes behind them.

The participants are all well equipped with the labs and machinery, not to mention the flow of patients, to contribute to the pool of data, but some are contributing more than others. Supported with philanthropic money that subsidizes its data efforts, Sloan Kettering sequences 410 genes from the tumor of some of its patients—mainly those with metastatic cancer. Dana-Farber looks at 405 genes, but it does so for every cancer patient. More financially constrained, Hopkins sequences about 50, and hopes to double that in the future, says Velculescu, but it relies on insurance and reimbursement to pay for its data work.

These disparities are a reminder that, while the cost of sequencing has plummeted in the genomic age, there are still financial limits to how much an institution can do. The differences also underscore a practical problem: How to combine all these data into one searchable pool? Not only are the data sets different sizes, but each institute might sequence a particular gene in different ways—focusing on one mutation over another, say, or sequencing the same site with varying levels of redundancy for quality control.

To help create a framework, GENIE has turned to Sage Bionetworks in Seattle, a nonprofit group that helps groups share scientific data. Part of the job is not just melding together existing data but accounting for future changes in each institution’s panels, or sets of genes, under examination. “These panels have to be living entities, driven by what the scientific … Next Page »

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